Sickle cell
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
Clinical News
January 22, 2021
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2…
EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
Clinical News
November 5, 2020
EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent Vaso-Occlusive Crises (VOCs) in patients, 16 and older, with sickle cell disease (SCD). This approval follows a recommendation for conditional approval issued in July…
Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU
Clinical News
October 2, 2020
Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU
Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high…
Coronavirus Disease among Persons with Sickle Cell Disease
Clinical News
July 13, 2020
Coronavirus Disease among Persons with Sickle Cell Disease
Individuals with sickle cell disease (SCD) are a particularly vulnerable group of patients, with a higher risk of severe complications due to COVID-19 infection than the general public. A recent…
GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Clinical News
July 7, 2020
GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease…
COVID-19 & Haemoglobin Disorders: The Impact of Coronavirus on Sickle Cell Disease Patients
COVID-19 Webinars
June 19, 2020
COVID-19 & Haemoglobin Disorders: The Impact of Coronavirus on Sickle Cell Disease Patients
An Expert Panel of Six Medical Professionals from various countries, such as India, the UK, Italy and Saudi Arabia, shared their experiences with TIF on the impact of the COVID-19…
COVID-19 & Haemoglobin Disorders: Compilation of Information for Sickle Cell Disease (2020)
TIF Publications
May 27, 2020
COVID-19 & Haemoglobin Disorders: Compilation of Information for Sickle Cell Disease (2020)
This document, prepared by the Thalassaemia International Federation , is a compilation of information exclusively for Sickle Cell Disease patients with regard to the coronavirus pandemic.
COVID-19 & Haemoglobin Disorders: A Classification of Risk Groups & Other Considerations (2020)
Publications
May 16, 2020
COVID-19 & Haemoglobin Disorders: A Classification of Risk Groups & Other Considerations (2020)
A TIF-compiled document aiming to provide a division of risk levels for thalassaemia and SCD patients with regards to the ongoing COVID-19 pandemic.
FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD
Clinical News
May 16, 2020
FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD
CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX-001, an investigational, autologous, Gene Editing hematopoietic stem cell therapy, for the treatment of severe Transfusion-Dependent β-Thalassaemia (TDT) and Sickle…
FT-4202 Granted FDA Fast Track, Rare Pediatric Disease Designations for SCD
Clinical News
March 2, 2020
FT-4202 Granted FDA Fast Track, Rare Pediatric Disease Designations for SCD
FT-4202, a potential disease-modifying oral treatment for sickle cell disease being developed by Forma Therapeutics, has been given fast track and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). A fast track status…